BioMarin Pharmaceutical Inc. a yau ya sanar da buga sakamakon daga binciken na 3 GENEr8-1 na valoctocogene roxaparvovec, wani binciken bincike na kwayoyin halitta don kula da manya da hemophilia mai tsanani, a cikin New England Journal of Medicine (NEJM). Labarin mai suna, "Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A," ya ba da rahoton shekara guda ko fiye na bayanan biyo baya daga binciken kuma an yi nuni da shi a cikin editan da aka buga a cikin wannan fitowar ta Jarida ta yarda da yuwuwar fa'idar zubar da jini da kuma gujewa. da yin amfani da prophylactic far.
Labarin binciken na asali ya ba da rahoton cewa biyo bayan jiko ษaya na mahalarta valoctocogene roxaparvovec sun sami raguwar adadin yawan zubar jini na shekara-shekara, rage yawan amfani da factor VIII, da haษaka aikin factor VIII, fiye da yadda suka yi a cikin shekara kafin yin rajista. A cikin ฦayyadaddun ฦayyadaddun adadin da aka ฦayyade wanda ya ฦunshi mahalarta 112 da aka yi rajista daga binciken da ba na gaba ba, yana nufin abubuwan da aka yi amfani da su na shekara-shekara na VIII da kuma ma'anar yawan zubar da jini bayan mako 4 ya rage bayan jiko da 99% da 84%, bi da bi (duka P<0.001) . Gabaษaya, mahalarta 121/134 (90%) ba su da ko dai ba su da jinni da aka yi da su ko kuma ฦarancin jinni da aka bi da su bayan jiko, idan aka kwatanta da prophylaxis factor VIII kamar yadda aka rubuta a cikin binciken da ba na tsaka-tsaki ba. A makonni 49-52, 88% na mahalarta suna da matsakaicin factor VIII ayyuka na 5 IU / dL ko mafi girma, kamar yadda aka auna ta amfani da chromogenic substrate (CS).
"Zin jini mai tasowa yana wakiltar babban nauyin kula da cututtuka da kuma rashin biyan bukatar likita ga mutane da yawa. An ฦarfafa ni cewa a cikin shekarar farko na jiyya, 90% na mahalarta nazarin sun sami ko dai sifili da aka bi da jini ko kuma ฦananan jini da aka bi da su bayan jiko fiye da factor VIII prophylaxis, "in ji Margareth C. Ozelo, MD, PhD, Darakta, Hemocentro UNICAMP. Jami'ar Campinas da Jagoran Babban Mai Binciken Nazarin GENER8-1. "Wadannan sakamakon suna nuna yuwuwar ci gaba da sarrafa zubar jini na hemostatic tare da wannan jigon jiyya na hemophilia A."
"Muna alfaharin zama majagaba a cikin nazarin ilimin halittar jini na hemophilia A mai tsanani da kuma raba bayanan marasa lafiya na kowane mutum wanda ke ba da damar fahimtar cikakkun bayanai game da cikakkun bayanai na wannan maganin da zai iya canzawa," in ji Hank Fuchs, MD, Shugaba na Duniya. Bincike da Ci gaba a BioMarin. "An yi nazarin Valoctocogene roxaparvovec fiye da kowane nau'in ilimin halittar jini na hemophilia A, kuma kowace shekara, muna ci gaba da ฦara iliminmu game da yadda wannan maganin binciken zai iya amfanar rayuwar mutanen da ke fama da hemophilia A. Muna godiya ga mahalarta binciken. da masu bincike don muhimmiyar rawar da suke takawa a cikin wannan shirin haษakawa, wanda ya haษa da GENEr8-1, mafi girman nazarin ilimin halittar jini a cikin hemophilia A."
Valoctocogene Roxaparvovec Tsaro
Wannan shine mafi yawan bayanan aminci na yanzu daga nazarin shekaru biyu na nazarin Mataki na 3 GENEr8-1 kuma yana rufe gabaษayan amincin valoctocogene roxaparvovec. Amincin da aka haษa a cikin littafin NEJM ya dogara ne akan nazarin shekara guda. Duk mahalarta a cikin binciken Phase 3 sun sami kashi 6e13 vg/kg guda ษaya. Babu mahalarta da suka haษaka masu hanawa zuwa Factor VIII, malignancy, ko abubuwan da suka faru na thromboembolic. A cikin shekara ta biyu, ba a sami sabbin siginonin aminci da suka fito ba, kuma ba a sami rahotannin muggan abubuwan da suka shafi jiyya ba (SAE). Yawancin marasa lafiya sun dakatar da duk wani amfani da corticosteroid (CS) a cikin shekara ta daya, kuma babu wani CS da ke da alaka da CS a cikin sauran marasa lafiya da aka cire CS a cikin shekara ta biyu. Gabaษaya, mafi yawan abubuwan da ba su da kyau (AE) waษanda ke da alaฦa da valoctocogene roxaparvovec sun faru da wuri kuma sun haษa da jiko na wucin gadi da ke hade da halayen haษari da haษaka mai sauฦi zuwa matsakaici a cikin enzymes na hanta ba tare da dadewa na asibiti ba. Alanine aminotransferase (ALT) haษaka (masu halartar 119, 89%), gwajin gwajin aikin hanta, ya kasance mafi yawan AE. Sauran abubuwan da suka faru na yau da kullun sun hada da ciwon kai (masu halartar 55, 41%), arthralgia (masu halartar 53, 40%), tashin zuciya ( mahalarta 51, 38%), haษakar aspartate aminotransferase (AST) ( mahalarta 47, 35%), da gajiya (40) mahalarta, 30%). A cikin binciken 1/2 na Phase, an gano wani SAE na ฦwayar salivary gland a cikin mahalarta binciken daya, wanda aka bi da shi fiye da shekaru biyar da suka wuce, kuma an ruwaito shi a matsayin wanda ba shi da alaka da valoctocogene roxaparvovec da mai binciken. An sanar da hukumomin lafiya da abin ya shafa a ฦarshen 2021, kuma duk karatun yana ci gaba da gudana ba tare da gyara ba. Kwamitin Kula da Bayanai mai zaman kansa (DMC) ya kara duba lamarin. Ana gudanar da nazarin kwayoyin halitta kamar yadda aka ฦayyade a cikin ka'idar gwaji na asibiti.
GENER8-1 Bayanin Nazari
Nazarin Phase 3 GENER8-1 na duniya shine nazarin hannu guda ษaya, buษaษษen lakabi yana kimanta inganci da amincin valoctocogene roxaparvovec a cikin mutanen da ke fama da hemophilia mai tsanani (FVIII โค 1 IU / dL) waษanda aka ci gaba da bi da su tare da prophylactic exogenous factor VIII. aฦalla shekara ษaya kafin shiga. Mahimmin mahimmanci na ฦarshe shine canji daga asali a cikin aikin factor VIII (CS assay) a makonni 49-52 bayan jiko. ฦarshen ingantaccen sakamako na biyu sun haษa da canji daga asali a cikin amfani da shekara-shekara na factor VIII tattarawa da kuma yawan adadin zubar jini na shekara-shekara bayan mako 4. An ฦididdige aminci ta hanyar rikodin abubuwan da ba su da kyau, gwajin gwaji, da gwajin jiki. Gabaษaya, jimlar mahalarta 134 sun karษi jiko na valoctocogene roxaparvovec guda ษaya a cikin adadin 6e13 vg/kg, kuma duk mahalarta suna da ฦarancin watanni 12 na biyo baya a lokacin yanke bayanan. An shigar da mahalarta 22 na farko kai tsaye a cikin binciken na Phase 3, 17 daga cikinsu ba su da cutar HIV kuma an yi su aฦalla shekaru 2 kafin ranar yanke bayanan. Sauran mahalarta 112 (yawan juzu'i) sun kammala aฦalla watanni shida a cikin wani binciken da ba na tsaka-tsaki ba don tantance yanayin zubar jini, amfani da fa'idar VIII, da ingancin rayuwa mai alaฦa yayin da ake karษar prophylaxis na factor VIII kafin a juye da karษar guda ษaya. jiko na valoctocogene roxaparvovec a cikin binciken GENER8-1.
